FDA Approves Drug for Severe Itching in Patients Liver Disease

FDA has approved Bylvay (odevixibat), the first treatment for pruritus (moderate to severe itching) in patients aged three months and older with progressive familial intrahepatic cholestasis (PFIC), a rare progressive liver disease that typically leads to liver failure.

In a healthy liver, cells drain bile acids into bile (a fluid that aids digestion), and the bile acid is removed from the liver. However, in patients with PFIC, liver cells are unable to drain bile acids into the bile, and these toxic substances build up in the liver. The exact cause of itching in patients with PFIC is unknown; however, it may be caused by increased levels of bile acids in the body and skin. Itching in patients with PFIC can be severe and can result in bleeding, excoriations (skin picking), scars, and discomfort that impacts daily activities and sleep.

PFIC usually appears within the first few months of a person’s life and affects an estimated 1-2 people per 100,000. There are at least three types of PFIC; all are inherited genetic conditions caused by gene mutations.

Bylvay is a reversible inhibitor of the ileal bile acid transporter (IBAT) and decreases the reabsorption of bile acids from the small intestines. Although the complete mechanism by which Bylvay improves itching in PFIC patients is unknown, it may involve slowing or stopping bile acid absorption by blocking the IBAT.